Novel transferrin receptor-mediated enzyme replacement therapy efficiently treats myogenic and neurogenic aspects of Pompe disease in mice.
新型转铁蛋白受体介导的酶替代疗法可有效治疗小鼠庞贝病的肌源性和神经源性方面
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2025.101547
George Kelly, Riley Raquel, Zhou Shan, Allen Elizabeth, Smith Laurie, Kistanova Elena, Kinton Sofia, Guo Lilu, Bangari Dinesh, Ismail Ayman, Thummapudi Jayaprakash, Boumezbeur Fawzi, Selingue Erwan, Tamarelle Dorothée, Capdevila Cecile, Do Tuan-Minh, Lesuisse Dominique, Leksa Nina, Sardi Pablo, van der Flier Arjan
